You might think of the ABCs as a fun, simple-to-learn childhood game. But those three letters signify a group of proteins with mutations that destroy functions in multiple organs, and investors are pumping $100 million into Rectify Pharmaceuticals to modify genetic diseases.
And the Cambridge, Massachusetts, biotech is founded by somebody who knows his ABCs: Chief Scientific Officer Jonathan Moore, Ph.D.
As a founding scientist at Vertex Pharmaceuticals, Moore worked on treatments that targeted the ABC transporter family. The Jackson 5 cheerfully sang "ABC," but this family of proteins is not a delight. The ABC transporters comprise 48 membrane-bound proteins with mutations that cause diseases in the lungs, liver, gastrointestinal tract, eyes and central nervous system.
During his nearly 28-year career at Vertex, until 2018, Moore worked on various treatments looking at the protein CFTR, which is part of the ABC family. The FDA approved multiple Vertex cystic fibrosis treatments targeting that protein, including most recently Trikafta in 2019 and Symdeko in 2018.
Now, Moore is the lead scientist at Rectify, which he founded the same year he left Vertex, according to his LinkedIn profile. The biotech officially launched with Atlas Venture last year and emerged Thursday with the series A funds from that VC shop, Omega Funds, Forbion and Longwood Fund.
“Outside of CFTR, there are currently no approved therapies that restore ABC transporter function, addressing the underlying cause of disease. Rectify is expanding the application of this proven approach to provide disease-modifying therapies for people living with serious genetic diseases caused by loss-of-function mutations,” Moore said in a statement Thursday.
The biotech is led by Rajesh Devraj, Ph.D., president and CEO and a venture partner at Atlas. Devraj was previously chief scientific officer at Disarm Therapeutics and, before that, president and CEO of Euclises Pharmaceuticals.
The C-suite also includes Chief Operating Officer Tracey Dawson, Ph.D., who came from the chief commercial officer post at Glympse Bio. Dawson has jumped from biopharma to biopharma with stints at Biogen, Shire, Genentech, Roche, Novartis and Eli Lilly.